This week on The Genetics Podcast, Patrick is joined by Chris Hopkins, CEO of Glafabra Therapeutics. They discuss how Glafabra is advancing a next-generation cell therapy for Fabry disease, the differences between gene therapy modalities, and the future of cell and gene therapy innovation.

Show Notes: 

0:00 Intro to The Genetics Podcast

00:59 Welcome to Chris

01:28 Overview of Fabry disease and reviving a promising shelved gene therapy

03:12 Limitations of current Fabry disease treatments versus Glafabra Therapeutics’ approach

05:19 How autologous cell therapy avoids an immune response

06:43 Comparing this cell therapy approach to that of Casgevy for sickle cell disease

11:28 Expanding Glafabra’s platform to other lysosomal storage disorders through enzyme cross-correction

13:47 Comparing autologous cell therapy and AAV in Fabry

17:02 Path to clinical development and funding strategy for Glafabra’s lead program

19:33 Cost efficiency and trial design advantages of an orphan drug approach

21:19 Considerations around comparator groups for Glafabra’s therapy

24:11 Underdiagnosis and hidden prevalence of rare diseases

25:53 Other innovations Chris is excited about and expectations for the future of cell and gene therapy

31:56 How Chris invented a technique to safely “milk” venomous cone snails

37:00 Closing remarks and advice for scientists taking the leap from academia to entrepreneurship

Find out more

• Glafabra Therapeutics (https://www.glafabra.com/)

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